Demographical and clinical description of 203 pES patients, and the study group of 80 patients
| . | pES patients, N = 203 . | Study group, N = 80 . | Others, N = 123 . | P, study group vs others . |
|---|---|---|---|---|
| Median age at first episode of cytopenia (min-max), y | 5.9 (0.2-17.4) | 5.9 (0.3-15.6) | 5.9 (0.2-17.4) | |
| Median age at ES diagnosis (min-max), y | 9.1 (0.2-19.4) | 8.7 (0.3-17.9) | 9.5 (0.2-19.4) | |
| Sex ratio (female/male) | 0.8 (91/112) | 0.68 (32/48) | 0.90 (59/64) | |
| Consanguinity, % (n) | 9 (16/177) | 13 (11) | 5 (5) | .047 |
| Immune manifestations in first-degree relatives, % (n) | 31 (57/181) | 30 (24) | 32 (33) | |
| Immune manifestations in extended relatives, % (n) | 38 (69/181) | 42 (34) | 37 (37) | |
| Occurrence of ITP and AIHA | ||||
| Simultaneous, % (n) | 42 (85) | 40 (32) | 43 (53) | |
| Sequential, % (n) | 58 (117) | 60 (48) | 57 (69) | |
| AIHA first, % (n) | 23 (47) | 14 (11) | 30 (36) | .01 |
| ITP first, % (n) | 35 (70) | 46 (37) | 27 (33) | .004 |
| Immune neutropenia, % (n) | 32 (64) | 44 (35) | 24 (29) | .002 |
| Associated immunopathologic manifestations, % (n) | 75 (153) | 82 (66) | 71 (87) | |
| Need for second-line therapy, % (n) | 72 (146) | 75 (60) | 70 (86) | |
| Rituximab, % (n) | 44 (90) | 51 (41) | 40 (49) | |
| Splenectomy, % (n) | 19 (39) | 17 (14) | 20 (25) | |
| Median follow-up from first cytopenia (min-max), y | 7.9 (0.1-29.0) | 9.1 (0.2-26.6) | 7.1 (0.1-29.0) | |
| Median follow-up from ES diagnosis (min-max), y | 5.8 (0.1-29.1) | 6.7 (0.2-25.0) | 5.5 (0.1-29.1) | |
| Deaths, % (n) | 10 (21) | 7 (6) | 12 (15) |
| . | pES patients, N = 203 . | Study group, N = 80 . | Others, N = 123 . | P, study group vs others . |
|---|---|---|---|---|
| Median age at first episode of cytopenia (min-max), y | 5.9 (0.2-17.4) | 5.9 (0.3-15.6) | 5.9 (0.2-17.4) | |
| Median age at ES diagnosis (min-max), y | 9.1 (0.2-19.4) | 8.7 (0.3-17.9) | 9.5 (0.2-19.4) | |
| Sex ratio (female/male) | 0.8 (91/112) | 0.68 (32/48) | 0.90 (59/64) | |
| Consanguinity, % (n) | 9 (16/177) | 13 (11) | 5 (5) | .047 |
| Immune manifestations in first-degree relatives, % (n) | 31 (57/181) | 30 (24) | 32 (33) | |
| Immune manifestations in extended relatives, % (n) | 38 (69/181) | 42 (34) | 37 (37) | |
| Occurrence of ITP and AIHA | ||||
| Simultaneous, % (n) | 42 (85) | 40 (32) | 43 (53) | |
| Sequential, % (n) | 58 (117) | 60 (48) | 57 (69) | |
| AIHA first, % (n) | 23 (47) | 14 (11) | 30 (36) | .01 |
| ITP first, % (n) | 35 (70) | 46 (37) | 27 (33) | .004 |
| Immune neutropenia, % (n) | 32 (64) | 44 (35) | 24 (29) | .002 |
| Associated immunopathologic manifestations, % (n) | 75 (153) | 82 (66) | 71 (87) | |
| Need for second-line therapy, % (n) | 72 (146) | 75 (60) | 70 (86) | |
| Rituximab, % (n) | 44 (90) | 51 (41) | 40 (49) | |
| Splenectomy, % (n) | 19 (39) | 17 (14) | 20 (25) | |
| Median follow-up from first cytopenia (min-max), y | 7.9 (0.1-29.0) | 9.1 (0.2-26.6) | 7.1 (0.1-29.0) | |
| Median follow-up from ES diagnosis (min-max), y | 5.8 (0.1-29.1) | 6.7 (0.2-25.0) | 5.5 (0.1-29.1) | |
| Deaths, % (n) | 10 (21) | 7 (6) | 12 (15) |
max, maximum; min, minimum.